Zoeken
The impact of vanishing white matter on unaffected family members
August, 2025
Romy J. van Voorst, Daphne H. Schoenmakers, Irene van Beelen, Francesco Gavazzi, Alexandra Chapleau, Adeline Vanderver, Geneviève Bernard, Ingeborg Krägeloh-Mann & Marjo S. van der Knaap
Orphanet J Rare Dis 20, 456 (2025). https://doi.org/10.1186/s13023-025-03987-8
Core protocol development for phase 2/3 clinical trials in the leukodystrophy Vanishing White Matter: a consensus statement by the VWM consortium and patient advocates
August, 2023
Daphne H. Schoenmakers, Prisca S. Leferink, Adeline Vanderver, Joshua L. Bonkowsky, Ingeborg Krägeloh-Mann, Geneviève Bernard, Enrico Bertini, Ali Fatemi, Brent L. Fogel, Nicole I. Wolf, Donna Skwirut, Allyson Buck, Brett Holberg, Elise F. Saunier-Vivar, Robert Rauner, Hanka Dekker, Pieter van Bokhoven, Menno D. Stellingwerff, Johannes Berkhof & Marjo S. van der Knaap
BMC Neurol 23, 305 (2023). https://doi.org/10.1186/s12883-023-03354-9
Therapy Trial Design in Vanishing White Matter - An Expert Consortium Opinion
April, 2022
Marjo S. van der Knaap, Joshua L. Bonkowsky, Adeline Vanderver, Raphael Schiffmann, Ingeborg Krägeloh-Mann, Enrico Bertini, Genevieve Bernard, Seyed Ali Fatemi, Nicole I. Wolf, Elise Saunier-Vivar, Robert Rauner, Hanka Dekker, Pieter van Bokhoven, Peter van de Ven, Prisca S. Leferink
Neurol Genet Apr 2022, 8 (2) e657; DOI: 10.1212/NXG.0000000000000657
Childhood Ataxia with Central Nervous System Hypomyelination / Vanishing White Matter
February 2003
van der Knaap MS, Fogli A, Boespflug-Tanguy O, Abbink TEM, Schiffmann R.
GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2020. PMID: 20301435 Bookshelf ID: NBK1258
Leukoencephalopathy with vanishing white matter: a review
October, 2010
Bugiani M, Boor I, Powers JM, Scheper GC, van der Knaap MS
J Neuropathol Exp Neurol; 69(10):987-96. doi: 10.1097/NEN.0b013e3181f2eafa. PMID: 20838246.
Vanishing white matter disease
June 29, 2021
van der Knaap MS, Pronk JC, Scheper GC
Lancet Neurol. 2006 May;5(5):413-23. doi: 10.1016/S1474-4422(06)70440-9. PMID: 16632312.